About this Author

College chemistry, 1983

The 2002 Model

After 10 years of blogging. . .

Derek Lowe, an Arkansan by birth, got his BA from Hendrix College and his PhD in organic chemistry from Duke before spending time in Germany on a Humboldt Fellowship on his post-doc. He's worked for several major pharmaceutical companies since 1989 on drug discovery projects against schizophrenia, Alzheimer's, diabetes, osteoporosis and other diseases.
To contact Derek email him directly: derekb.lowe@gmail.com
Twitter: Dereklowe

February 10, 2010

Where Would You Start a Company?

Posted by Derek

We've been talking a lot around here about small companies versus large ones, the merits of different therapeutic areas, and so on. So here's a question: if you were starting a small drug company today, where would you concentrate its efforts?

Oncology? Ten years ago, you could make that case, I think. But now everyone's piled into the area, so you'd have to have a real edge to make a go of it. For one thing, finding patients for clinical trials is a major problem. Your best shot here would be really obscure varieties of cancer, I'd think, unless you've got something really major. And how do you ever know if you've got something really major or not in this area until you get to the clinic, anyway?

Anti-infectives? There's certainly room for some new niche products here, but that's what they're going to be. And this is a surprisingly difficult area to make headway in, if you haven't worked in it before. Nothing's going to be an almighty blockbuster here (because nothing new is going to be a frontline therapy), but there is money to be made.

Cardiovascular and metabolics? I don't see how, and I barely see why, unless you've got the miracle HDL-raising pill up your sleeve. Diabetes, for its part, has been a fine area over the last ten or twenty years, but the safety criteria for a new therapy are now very stiff (and the market is pretty well covered, from several different angles). Not recommended, I'd say.

Alzheimer's? Good luck! Man, is there ever an unserved market here, but it's unserved for a lot of damned good reasons. The same goes for a number of other CNS indications. This whole area is a tightrope of risk and reward. Both are huge.

Or would you go the Genzyme route, making huge amounts by helping out people (a few people) that no one else can help at all? Again, this presupposes that you have some really good idea about how to approach these orphan diseases, and it's going to be tough to make a whole company out of them (since they're spread over such disparate therapeutic specialties). But this would seem to be feasible, with some luck.

Suggestions are welcome in the comments. I'm definitely not planning on starting a company myself, but I think that we need as many as possible, and perhaps some ideas will trigger something for someone in a position to act. . .

I think that starting a company to help pharma management outsource their R&D operations is the way to go. Every MBA and lawyer is trying to outdo the other sociopaths..

Think about it.. you get lots of money for pretending to discover stuff, and within five years you can cash out and fire all your employees. Oh wait.. that is just a modification of the current CRO and Biotech model.

Seriously.. has business been ever able to predict a blockbuster in advance? as in EVER? Read some history books to see what people thought of cell phones, PCs, microwave ovens, rocketery, anti-bacterials, anti-depressants (pretty much every single innovation). Conventional wisdom in business is always incapable of understanding innovation.

Best of luck following these ivy league shysters, because if you believe them- you deserve them!

I would start a company directed at regulating the autoimmune response to EBV and EBV-induced antigens in multiple sclerosis and lupus. The epidemiology is so strong associating that virus to those two diseases that the link is essentially incontravertible at this point. Finally, the patients would have a company targeting the source of the inflamation rather than having their doctors suggest cyclophosphamide treatments or trying to enlist them in a clinical trial targeting the same (wrong) autoantigen that has already failed in 10 different ways.

If you are a VC group out there, I've got 25 years of (successful) Med. Chem. experience and I know the virologists and biologists. We can all get rich together!

One could argue for diseases of aging being ripe: age-related macular degeneration, Parkinson's and Alzheimer's, ALS, etc.

But the markets no one seems much interested in are birth control and pregnancy. There is almost no competition in the latter and the former has really seen only a couple of new entrants during the past two decades. Unless people change their attitudes towards sex, there will be an on-going, and expanding, market for birth control.

One other thing: from a business model perspective, I'd set the company up to take the drug/biological/vaccine (if anyone is interested in vaccines at this point) to the EOP2 (the End of Phase 2) meeting with FDA. That requires a minimal amount of capital. It's the Phase 3 studies that cost a fortune. Better to leverage and sell the product to a big pharma house after the EOP2 meeting. You could probably do that for under $20 million, maybe even under $10 million. Of course, that also assumes that management understands something about developing a drug, as in not trying to pawn an uncontrolled Phase 1 study as a POC demonstration.

To #5: we should all live so long as to see competent managements in the pharmaceutical industry. I don't think we will see that for the rest of our lifetimes. But that there was another Vagelos and Skolnick running a pharma company.

Don't you guys get it! The system has evolved into something that disdains innovation, and not just in pharma. Until the flawed system is allowed to die from it's own shortcomings, nothing will change. Just hope that it will eat itself quicker than it already is.

Remember that we may be very innovative, but the world listens to priests from ivy league business and law schools. Unless these priests are discredited by their actions, people will never believe you. It took the black death to usher in the renaissance..f

Pharmacoepidemiologist- I agree with you on the birth control/pregnancy area. A non-hormonal birth control pill would be huge. Bonus points if it is safe to use while breastfeeding. Just because people are using the existing options, it doesn't mean that they are HAPPY with them. I can personally tell you that your birth control options while breastfeeding just downright suck.

And the treatments available for most causes of infertility are apparently even worse.

I also think there is room for better treatments in most autoimmune diseases, including asthma and allergy.

Regardless, this isn't really how most new start ups get started. They tend to start from a technology and then go looking for a therapeutic area in which they might be able to apply it. I've often wondered if someone could make a success of taking a more "traditional" drug discovery approach and just starting a new company. I suspect they'd have a hard time convincing the VCs to fund them without some hook that is supposed to be their competitive advantage.

You're all wrong. Most of the above maladies result from aging. Concentrate on slowing or eliminating the aging process will give society the biggest bang for their buck. I mean jesus, there are only like 3500 'druggable' targets in the human body. You need to get to the source of the problem.

Just off the top of my head, I would concentrate on areas that still may have therapies, but not necessarily good ones, like asthma, COPD/emphysema, chronic pain and the like. Things that may soon affect me as I age, but which also affect others in the general population.

In a perfect world, the trick I believe is finding the right biological targets and having the right expertise in house at the small company. You can typically execute the discovery to get to proof of concept, if the resources are there. It's the thousand other political, etc things that tend to have the most damaging effects. And, you have to have a good, focused strong leader in charge.

Not to squelch Greg Hlatky (#2), but you need to distinguish between two kinds of veterinary pharmaceuticals: those for companion animals (cats, dogs, etc.) where you can presumably charge a premium in the same way you can for human pharmaceuticals, and those for "farm animals" (cows, sheep, etc. - I forget the conventional term), where cost-effectiveness is key. No farmer will pay more to cure a cow than it costs to replace it, but there are a number of people who will pay thousands to prop up an aging/sick dog or cat.

Heck even though you asked about small "drug" company I would set up a Neutraceuticals operation. Start-up and maintenance costs would be substantially lower and then could make claims about treating all the things mentioned and don't have to prove any of it. Only fault is how to capture a share from all the competitors that are in that line now. Any recommendation on a catchy Company Name/Logo?

1) To grap market share, how about sponsoring an extreme sports tour or a rock concert series? Another option: get a sports coach to be a pitchman. Jimmy Johnson's taken, but maybe you can get Marty Schottenheimer. Eric Mangini should be available in a year or so.

Love this question, and have thought a lot about it over the last three years, from both sides of the table.

First, I think the question is worthy of discussion, and interesting, but practically, upside down. I think it is a common mistake to think of unmet need as a driver of value, but it is your technology, your molecules that determine your path and success on that path. In the old days, like the 80s, companies were started with beliefs that good things could be done in areas of need, but not today. In the interim years, VCs loved platform plays because they had more chances of success. However, most of these had to make a transition to therapeutics company after the platform buzz wears off, and few do it successfully. Today, not much is funded, and though there is some variation, best to be peddling a molecule with sellable attributes. Got to feed the pharma pipeline!

For industry scientists, this molecule-in-hand demand creates a real chicken-egg problem. Unless your current company will let you walk with IP, you've got nothing but know-how, which though in short supply in the VC hype world, is worth about a nickel.

Last note, the most common pitch I saw when working with VCs was, to paraphrase, cancer is really bad, look at this target in mouse KOs, so all we have to do is make a drug. Got 10MM? I got to the point where I just about puked when someone spent time detailing unmet need. The more they did that the more I knew they had nothing in terms of a real lead molecule, and no sense of how to get there, and often no idea of industry efforts that littered the ground with failures.

But that said, if I could pick an area, I would pick autoimmune/inflamm. Many targets, some of which may also play in cancer if the safety profile is not suitable for things like asthma.

Thought of the vet side but saw data that said the economics are tough. Most people have about a $500.00 cut off for pet therapy. Above that, the market shrinks as people elect to forgo therapy or euthanize.

On the neutraceutical side, my favorites were the cookie diet, since I love cookies, but someone did it, then the color diet, complete with personal color palates, all missing white/yellow to reduce calaories, but someone did that one too! How about Pizza and Beer? Have a beer for breakfast, another for lunch, then our mail order Pizza for dinner. Like every other stupid diet, just keep the calories to 1400/day and people will lose weight.

Derek BTW I did not mean to trivialize a serious post question but is truly what I first thought of.

#16 Hap extreme sports and rock concert might hit the right section of gullible demographic although wouldn't the sports figures mentioned be more on the geriatric side (so I would go for PSU coach JoPa?

Natural High perhaps if was based in Rocky Mountains although I think this used to be part of an "anti-drug slogan".

#17 Cellbio is Pizza and Beer no longer a customary breakfast for most college kids these days?

anti-infectives face one barrier that no other disease area faces. If you came through with a highly-effective antibiotic that hit resistant organisms, the FDA would approve it ONLY for last-line use to delay the development of resistance. You would totally own a very very very small market niche and would never make back your costs.

1) JJ is supposed to be promoting a male "enhancement" product common to men's magazines - I guess pounding the hell of it in men's magazines hasn't gotten them enough business from gullible men. I was sort of hoping that JoePa wouldn't go for that (my opinion of him isn't that low) - for the older demographic, I would maybe go for Bob Huggins (might also appeal to younger demographic in the "recovering from hangover" class), and maybe Neuheisel or USC's coach for younger ones. I'm not certain why coaches should be good ad people for such campaigns, but I don't know why these ads work at all. (Just because you have a problem doesn't mean the Russian spammers on the Internet or the companies running ads in the back of magazines have any idea how to solve it.)

If you really have decide that your conscience is unnecessary, you could go for the "toxic pharmaceuticals are bad for you" shtick. (Cleansing products "to remove toxins" would be a cheap start.) Jenny McCarthy might be available for that campaign. Cost of goods should be minimal, other than keeping them sterile.

2) CNS and cancer both seem open (when there are lots of ways to do something, that usually means that none are any good, as someone else said), but the problems with drugs seem to be, in a nutshell, that you don't know what you have until it's in the clinic and a billion dollars (or more) is spinning down the toilet. That seems like an area for platform technologies, but if they could have been done easily, well...

CNS is still a decent domain because the therapeutic domain is so systemically corrupt.

Look at the garbage drugs like Seroquel and Cymbalta. Dirtier than heck, but they sell billions both on and especially off label. FDA fines are chump change compared to sales.

And the clinical trials can be gamed, FDA is (mostly) indifferent, plenty of psychiatrists will pimp the product and the off-label market is enormous because mental illness diagnoses are so subjective.

CNS drugs in a lot of respects or are the snake oil "elixers" of the 21st Century. Still a lot of money there.

Vaccines for the pandemic each year. You know, SARS, H1N1, Swine flu, Bird Flu, West Nile Virus, EEE, they just keep coming. And then when very few people get sick, you claim it was because your preemptive treatment. Money for nothing.

Since I have always been employed as a medicinal chemist at big pharma, this is something that crosses my mind regularly. If I was in a financial position to take part in a startup company, I would choose either autoimmune disease, antibacterials, or pain. Here is why:

autoimmune disease - large market potential, many medicines to be discovered yet.

antibacterials - big pharma is mostly out of it, leaving room for the niche market. Even relatively small sales could be a big profit for a small company.

pain - relocate to california and start growing cannabis for "medical" use (this is also my backup self-employment strategy when I get made redundant)

Many med. chemists over here have criticized outsourcing as one way to potentially bring down the drug R&D cost. A bit self-serving I'd say since obviously this trend eliminates a lot of jobs here, which has seemed to pick up the speed and snow balled lately. For those who oppose outsourcing while now proposing to start and manage your own drug R&D adventure, honestly do you not intend to use these service firms to try to stretch your fund, assuming it's not as abundant as you'd like? Also assuming IP protection is not an issue.

The problem with nutraceuticals is that the FDA has really moved to shut down 'legitimate' marketing. For example, a company cannot link to an independent, NIH funded research study if that study mentions a clinical (disease-related) use. So Harvard did a study that showed that your magic ingredient cures cancer? Sorry... you can't even mention it because that is considered marketing for the treatment of cancer, and for that you have to go through the full NDA process. Although this may have been done in good faith, the real effect is to drive out legitimate information/companies, leaving the fly-by-night outfits to dominate the market. If you don't really care to toe the line, the typical two year delay in FDA enforcement is enticing to those on the dark side.

Second, the FDA has reserved the 'right' (although I haven't seen any specific examples yet) to declare that an ingredient is actually a drug (and therefore illegal to sell until it has gone through a NDA) if any clinical trial is registered with the goal of testing a clinical hypothesis. So even if you keep your nose clean, do everything by the book, and have a real product with good health prospects, some independent academic at Big State U can start a clinical trial, make noise and attract the attention of the FDA, and boom, you are shut down. Most, if not all, nutraceutical products don't have the potential market to support a full Phase III evaluation, so when the costs surge, they just go away.

I would put my money on making drugs for device-drug combos. Most device companies don't have much drug expertise, you can resurrect old drugs that are off-patent and/or were too toxic when taken through the GI, and the R&D should be somewhat shorter since existing data can be used.

Milkshake, hear from several VCs a growing interest in ocular disease. If that herd moves there, probably just means they don't yet know the challenges, that is they have no experience.

The selling points are growing market with the aging population (blockbuster), and you can repurpose existing molecules that made it into man but had safety issues that would not be a problem with local delivery. Not really a big research play.

The “differentiation” in terms of molecules and MOA is the key. Look no further than chronic pain (as mentioned earlier) where there are huge unmet needs, growing markets and practically no anagesics without side-effects/addition/tolerance. Besides, the clinical cost up to POC (P2) in pain is much cheaper, shorter and more predictable than cancer. The most apparent “do-able” start for the experts of med chem here may be to “have” a molecule with high subtype selectivity or even better with an “allosteric”, orally- and CNS-active inhibitor for some clinically (P2 or P3) validated new (kinase) targets, where antibodies or peptides have been validated in P2 or P3 human clinical trials but no small molecules yet. It is of course much harder (so the “differentiation”) to identify even an allosteric molecule, let alone orally- and CNS-active (or CNS-inactive if intended to target peripherally). An example of such target, NGF or better yet its receptor NTRK1 - Neurotrophic Tyrosine Kinase Receptor Type 1, aka TrkA (or related downstream signal transduction pathways). Any thoughts or comments? Any other such targets in pain or other disease areas?

As a primary care rep, I would like to offer a different angle: How about, What would doctors like to see me bring them? Something DIFFERENT! Nothing with a CR or XR attached to it, not another statin or glitazone, but a new class of drug for a common disease state that works better than what they have, something they can trust, something they can tell the managed care companies when they call to request generic use first: NO. They would also like pain drugs that worked and did not turn patients into drug seeking zombies calling with lame excuses as to why their 30 day script only lasted 10 days.

"honestly do you not intend to use these [CRO] service firms to try to stretch your fund, assuming it's not as abundant as you'd like? Also assuming IP protection is not an issue.

I'm just curious."

To PC: IP protection and quality are much bigger issues than people realize. I'd try to use CRO's only for intermediates, if possible, and I'd double check the quality! Failing that, I'd go North America, followed by EU, before resorting to Asia.

"As a primary care rep, I would like to offer a different angle: How about, What would doctors like to see me bring them? Something DIFFERENT! Nothing with a CR or XR attached to it, not another statin or glitazone, but a new class of drug for a common disease state that works better than what they have"

Tell that to the FDA. The latest C&EN has a damning indicted about FDA mishandling of anti-infective applications.Maybe they are filled up with Ex-Pfizer employees seeking revenge.

There is of course some irony in the regrets shown for pharma cutbacks to R&D while simultaneously struggling to determine attractive new areas to pursue.

What is still attractive? First place I'd think to look would be where is venture capital putting money? A quick glance (moneytree report) at the top five deals in Q4 in the New England show interest in pain, cancer vaccines, inflammation (particularly autoimmune), chronic infection, gastrointestinal, and others. Curiously, pharma risk management services was also on the list.

Another thing to consider, in an atmosphere of increasingly more stringent regulatory oversight, I'd be thinking of business opportunities that can in any way assist with the FDA's critical path initiative.

I am curious to hear what this group's response would be to the generic business model? Find some excess manufacturing capacity, a China company to produce your API and you are in business. Virtually no risk of refusal from the FDA and as long as you stay from the huge blockbusters you should make a living.

startup generic company in China? No thanks. Native chinese founded plenty of companies covering almost every economically sensible niche. Obviously if you plan to start a real generic company, file ANDA's and register DMF's things are a bit different but in this field the winning model is the illegal one: non inspected API plant on one side, a brokering company that fixes the papers in the middle, the final western vendor on the other side.
Also exporting technology to China is not smart at all.

startup generic company in China? No thanks. Native chinese founded plenty of companies covering almost every economically sensible niche. Obviously if you plan to start a real generic company, file ANDA's and register DMF's things are a bit different but in this field the winning model is the illegal one: non inspected API plant on one side, a brokering company that fixes the papers in the middle, the final western vendor on the other side.
Also exporting technology to China is not smart at all.

This thread is so long that I didn't read all the other replies. So this might have been proposed.

A male contraceptive

If you could actually get this to work, you would make so much money it would be ridiculous. Women don't like taking contraceptive pills. Some women can't take contraceptives. No one likes using condoms with their regular partner. Many (MANY) men would like to have some extra armor to make sure no one sneaks by and commits you to something you may not want for the next 18+ years.

Good luck though. The female reproductive cycle has periods of greater and lesser fertility and critical steps that can be disrupted by drugs. The male reproductive system is always "on". Your drug would have to be reversible. No one wants to be chemically castrated and never have kids because they wanted to sleep around in their 20's and not worry about condoms.

People reject lots of good ideas, but they also reject lots of bad ones as well - if people are that bad at being able to distinguish between good and bad ideas, well, then their beliefs shouldn't affect how I choose a field of interest. If you have an idea that everyone thinks is bad, there'll be less competition, but unless there's some good data, the probability of success is probably even lower than in a crowded, but more likely successful, field.

Bingo. Set up in China, hire some local employees, then watch as native competitors spring up using your tech, your ideas, and your methods. Industrial espionage of foreign companies is endemic over there, and more than likely government-assisted.

I would start a new company focusing on cancer products, antibiotics (preferably for gram negative organisms) and CNS disorders. The angle, however, would be to come up with novel ways to deliver these drugs so they would be organ specific, demonstrate improved pharmacokinetics or combined to produce synergistic pharmacological benefits. An example of this would be CarrierWave technology that Shire acquired when they purchased New River Pharmaceuticals several years ago.

When I finally get the bullett I'm gonna sell overpriced uniquely formulated hexapeptides (sourced from the orient) which would be gauranteed# to keep you looking young. With exquiste packaging and late night advertising campaigns I'm sure I couldn't fail.

Personally I'm planning on working on a cure for being ginger. If you think about it, it's the perfect disease - highly prevalent (4% of the european population), almost everyone knows one, it's chronic (no-one really recovers), frankly the rest of us find them rather repulsive and it's a single gene disorder of the melanocortin-1 receptor. Perfect. Anyone want to join me to make the world a ginger-free zone ?

Surprises me that nobody here commented on the obvious: if you're starting a small company, it better be in an area that can attract funding. Now, there's lots of ways for a small company to get funded, but by and large the most substantial and transformative is a classic venture capital investment. So-called 'angel' investors aren't interested in therapeutics, and the SBIR/STTR system is a mess. Disease foundations are doing some innovative things (e.g. the Alzheimer's Drug Discovery Foundation) but they are still few and far between. Nope, its still VC funding that gets small biotechs off the ground.

With that in mind, the VC community is currently 'overweighted' in cancer companies, so unless you've got a cancer drug that's on the cusp of phase 3, no funding for you. There are pockets of interest in anti-infectives (one of my own personal pet projects) but less so. Everyone wants to cure neurodegenerative diseases, but the recent high-profile clinical failures have everybody pretty darn leery, so compelling data is needed to get any attention at all. You're not going to get anywhere with a VC on a CVD indication, unless its really niche. Diabetes (esp. as an entrance to the massive obesity market) is pretty hot, but interestingly inflammation is hard to get traction on... the road is littered with failures in auto-immune diseases, and VCs always have a bad case of TCSYC (they couldn't so you can't... aka herd mentality). There were a few mentions above of ocular indications, and that was a hot investment area about 18months ago, but its trailing off because most VCs have made their bets already.

Recent fundings suggest that the hottest area is platform technology companies (yes, they are coming back into vogue) that are focusing the first clinical effort on orphan indications. Kind of Genzyme but with a technology (siRNA, peptoid, whatever) that can be applied to multiple therapeutic 'shots on goal' (and I hate that phrase by the way).

Unfortunately, there just aren't many new 'platform technologies' that could underpin these opportunities. Classic small-molecule-applied-to-disease approaches are all but ignored, as it forces the VC to bet that the single compound will be successful through Phase 2 (in their opinion the spot where they can reach an return on investment) and almost no VC I can think of is willing to make that bet. Combine this with an overall retraction in the VC market (VCs have their own investors and they are getting super skittish... VC shops are actually closing because *they* can't raise money for *themselves*), and it makes for a rough biotech funding marketplace.

Oh, and don't even bother with nutraceuticals, agrochemicals, or veterinary markets - all are essentially DOA on a VCs desk.

1. I'd start with good science. In several places I've worked, that was rather passe or worse: it was considered contrary to the officers' fiduciary responsibilities (to themselves, of course). Even now, a bogus business plan will beat out good research every time.

2. I'd mix it up, by which I mean: Sometimes I'd start with a good chemistry idea and run with it (towards a highly proprietary position) and see if it works in somebody's assay. Sometimes I'd start with a target and attack that; the compounds will end up in off target assays anyway.

3. I do not like the idea of trying to design for blockbusters or trillion dollar molecules. If something is good and useful, it will grow market share. I do like orphan disease targets.

4. Someone above mentioned vision. I agree and hope that we're both right.

I could elaborate on the above but who is going to read all the way to the end of 60+ comments?

For instance, go into generics - but with a few tweaks here and there, e.g. combination pills (beta-blockers + low-dose statin). Take a look at how Atripla has transformed first line therapy for HIV patients.

Take generic molecules and focus on "innovations" that improve drug delivery or half-life (as Abbott has done with Niaspan, and Abraxis with nab-paclitaxel).

Focus research on novel and safe pharmacoenhancers that could be combined with other molecules and formulated in a single pill. This is being done in HIV, but I don't see why this can't be pursued in other therapeutic areas as well. For instance, you could develop a $1/day pill that enhances the half-life of a $25/day pill - potentially requiring lower doses, and less cost to the patient.

> if you were starting a small drug company
> today, where would you concentrate its efforts?

Well, if I followed the old saw about making something I'd personally like to have, I think I'd have to focus on trying to make an OTC cold med, particularly a decongestant. There's really only one you can buy that's any good (pseudoephedrine), and it's a pain to buy these days because of the new restrictions on selling it. So most people are buying phenylephrine, which doesn't bloody work. (There's also guaifenesin, but all that does is make the mucus runnier, while INCREASING the quantity.) There's still mentholated cough drops, but they only get you so far. You can clear your nose that way, but not your whole head. A new decongestant that can be sold right off the shelf but actually *WORKS* would probably sell like hotcakes, if priced vaguely reasonably and advertised well.

Yeah, I know, there's not as much money in OTC as in prescription meds because people have to pay for it out of pocket directly instead of letting their employer's medical "insurance" company pay it out of what was deducted from their salary. So maybe it's a crummy idea. But it was the first thing that popped into my head.

>if you were starting a small drug company today, where would you concentrate its efforts?

That kind of depends. If for benefit of humankind, then probably oncology. If for raking in money, then lifestyle drugs - obesity, impotence.

Also it would be seriously influenced by what biologists and their expertise I could get, because, as sad as it might be, chemistry and med chem are somewhat secondary in creation of a drug to what your biologists do.

It's a tough road, but I would work on gene therapy treatments. I don't care if it's RNAi, gene replacement, whatever, but it seems to me that if any significant progress is going to be made in any genetic disease, then someone has to figure out the delivery, and it's only a matter of time before someone does. If I were starting a company, I'd want it to be me.

a "silencer" for latent viral suppression. throw some methyls in the mix and make it happen. if we "silence" or "hide" the protor region, nothing gets transcribed, no? when everything else fails, lets just hide the damn genes... of course, sensitivity may be an issue.

I'd go for birth control. Either of the ideas already mentioned (male BC or a non-hormonal female BC) would be excellent. Right now, there's only one group of pills for it, they only apply to one gender, and for some group of women (myself included) the side effects render them unusable.

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Currently out of the running Fortune 500 companies, there are only 16 female CEOs (as told by USA Today).

In today's pharmaceutical marketplace, whether mergers and acquisitions are causing reduced competition; or FDA actions against problem manufacturing facilities are interfering with production; or raw material shortages have limited the ability to produce adequate amounts of drug to meet demand....availability of some commonly used pharmaceuticals have become a problem or pricing has gone through the roof.

I would be inclined to develop a generic company which would initially focus on developing products in these higher demand areas where prices is currently elevated. In other words, I would let the demands and nuances of the pharmaceutical marketplace determine my initial product focus.

I would be inclined to initially use manufacturing, research and testing outsourcing with a responsible partners to produce the products in order to keep initial capitalization costs at a minimum.

I would put an above-average focus on marketing
with additional emphasis on appealing to pharmacy buyers and pharmacists....possibly developing a direct sales system. (Being a pharmacist and past pharmacy owner, myself, I see some opportunity here). I would seek to appeal to both the mass market and professional market. I
think a balance in this area could bring stability to the company's bottom line.
If necessary, I would use a different company name to appeal to each, separate market area (to avoid potential conflicts) [all under one umbrella company].

I would seek to establish competitive bid contracts for extended periods of 6 to 12 months with higher volume purchasers with a number of these higher-than-normal-priced products to establish a stable financial base for the company.

I would continue to expand product offerings into other products with similar, favorable market issues.

As sufficient, additional working capital is generated, the company would begin developing its own manufacturing, research and testing capabilities (if it was determined economically and/or logistically beneficial to do so).

Creative and effective dosage form designs and characteristics would be pursued.